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Novel RNA-targeted therapies for hereditary ATTR amyloidosis and their impact on the autonomic nervous system

PURPOSE: Transthyretin-mediated hereditary amyloidosis (hATTR amyloidosis) is a multisystemic disease with heterogeneous clinical presentation. Hallmarks of the disease are sensory-motor and autonomic neuropathy and cardiomyopathy. Two disease-modifying drugs, inotersen (an antisense oligonucleotide...

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Detalles Bibliográficos
Autor principal: Conceição, Isabel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6763402/
https://www.ncbi.nlm.nih.gov/pubmed/31399774
http://dx.doi.org/10.1007/s10286-019-00626-8